JP6756700B2 - 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 - Google Patents

筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 Download PDF

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JP6756700B2
JP6756700B2 JP2017501101A JP2017501101A JP6756700B2 JP 6756700 B2 JP6756700 B2 JP 6756700B2 JP 2017501101 A JP2017501101 A JP 2017501101A JP 2017501101 A JP2017501101 A JP 2017501101A JP 6756700 B2 JP6756700 B2 JP 6756700B2
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pharmaceutical composition
nucleic acid
c9orf72
cells
raav
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ミュラー，クリスチャン
ブラウン，ロバート，エイチ．，ジュニア
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ユニバーシティ オブ マサチューセッツ
ユニバーシティ オブ マサチューセッツ
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    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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JP2017501101A 2014-03-18 2015-03-18 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 Active JP6756700B2 (ja)

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US201461955189P 2014-03-18 2014-03-18
US61/955,189 2014-03-18
PCT/US2015/021321 WO2015143078A1 (en) 2014-03-18 2015-03-18 Raav-based compositions and methods for treating amyotrophic lateral sclerosis

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JP2019159514A Division JP7051118B2 (ja) 2014-03-18 2019-09-02 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法

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JP2017510298A JP2017510298A (ja) 2017-04-13
JP2017510298A5 JP2017510298A5 (enExample) 2018-04-26
JP6756700B2 true JP6756700B2 (ja) 2020-09-16

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JP2017501101A Active JP6756700B2 (ja) 2014-03-18 2015-03-18 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
JP2019159514A Active JP7051118B2 (ja) 2014-03-18 2019-09-02 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
JP2021093003A Pending JP2021152030A (ja) 2014-03-18 2021-06-02 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
JP2024000089A Pending JP2024032730A (ja) 2014-03-18 2024-01-04 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法

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JP2021093003A Pending JP2021152030A (ja) 2014-03-18 2021-06-02 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
JP2024000089A Pending JP2024032730A (ja) 2014-03-18 2024-01-04 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法

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US (6) US10280418B2 (enExample)
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AU (3) AU2015231294B2 (enExample)
CA (1) CA2942515C (enExample)
DK (1) DK3119797T3 (enExample)
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